DE PNH Patient Flow Model

Germany is the one market in this model family where the disease-burden layer starts with an explicit gap rather than a number. The DGHO's Onkopedia clinical guideline states plainly that Germany-specific PNH prevalence figures do not exist; the estimate it offers instead, 16 cases per million prevalence and 1.3 new diagnoses per million annually, is extrapolated from British and French registries, not counted domestically. Any epidemiology-based forecast for a German PNH launch inherits the uncertainty of that foreign-registry extrapolation rather than a domestic count. Diagnosis itself requires GPI-anchor flow cytometry, at least two GPI-anchored markers showing deficient or reduced expression across at least two cell lineages, typically granulocytes and reticulocytes, and concentrates at just two named national institutions: the German PNH-Register, housed at Ulm's Institute for Clinical Transfusion Medicine and Immunogenetics, and the West German Cancer Center at University Hospital Essen. Both feed the International PNH Registry, the same dataset underpinning the long-term ravulizumab and eculizumab outcomes literature, but neither publishes an independent German prevalence count of its own.

The treatment-eligible layer is better sourced than the epidemiology layer, because it runs through Germany's AMNOG appraisal process rather than a registry count. Iptacopan, as an orphan-designated therapy, had its additional benefit established through EMA approval alone under paragraph 35a of SGB V, so it skipped a comparator dossier entirely, and the G-BA still found a substantial additional benefit on quality of life on 19 December 2024. Ravulizumab's only PNH-specific G-BA review, a March 2022 assessment of its pediatric indication against eculizumab, found no additional benefit. Crovalimab submitted its benefit dossier on 12 September 2024 and remains under active IQWiG assessment, with no G-BA decision yet. On pricing, no negotiated net price for iptacopan has surfaced in the Lauer-Taxe as of this review, consistent with the manufacturer electing the 2025 MFG confidential-pricing mechanism, a gap with consequences for the 15 countries that reference Germany's published price in their own reimbursement formulas.

Every AXLRx patient flow model is built on a five-layer funnel: population, disease burden (E1), diagnosis and specialist capture (E2), treatment and biomarker eligibility (E3), market access (E4), then Year 1-3-5 projections across three scenarios. Delivered as a live Excel workbook, not a static table, across 8 sheets with zero hardcoded cells.

Germany PNH sources: the DGHO Onkopedia clinical guideline, the German PNH-Register (Ulm) and West German Cancer Center (Essen), G-BA Nutzenbewertungsverfahren decisions for iptacopan and ravulizumab, IQWiG's Dossierbewertung for iptacopan, and the GKV-Spitzenverband negotiation registry.